Abstract

Despite recent improvements, α1-antitrypsin deficiency (AATD) remains a rarely diagnosed and treated condition. To assess the variability of AATD diagnosis/treatment in Europe, and to evaluate clinicians’ views on methods to optimise management, specialist AATD clinicians were invited to complete a web-based survey.

Surveys were completed by 15 physicians from 14 centres in 13 European countries. All respondents perceived the AATD diagnosis rate to be low in their country; 77% of physicians believed that ∼15% of cases were diagnosed. Low awareness was perceived as the greatest barrier to diagnosis. Spirometry was considered more practical than quantitative computed tomography (QCT) for monitoring AATD patients in clinical practice; QCT was considered more useful in trials. AAT therapy provision was reported to be highly variable: France and Germany were reported to treat the highest proportion (∼60%) of diagnosed patients, in contrast to the UK and Hungary, where virtually no patients receive AAT therapy. Most clinicians supported self-administration and extended dosing intervals to improve convenience of AAT therapy.

This survey indicates that AATD diagnosis and management are highly heterogeneous in Europe; European cooperation is essential to generate data to support access to AAT therapy. Improving convenience of AAT therapy is an ongoing objective.

Abstract

Access to α1-antitrypsin (α1-AT) therapy varies in Europe; where available, α1-AT therapy optimisation is the goalhttp://ow.ly/YL6m30n4LV3

Conflict of interest: J. Chorostowska-Wynimko reports grants, personal fees, nonfinancial support and other support from Grifols and CSL Behring, personal fees from Kamada, and other support from Shire, outside the submitted work.

Conflict of interest: J.H. Ficker reports grants from CSL Behring during the conduct of the study; and personal fees from Grifols, and personal fees and non-financial support from CSL Behring, outside the submitted work.

Conflict of interest: G. Thabut reports personal fees and non-financial support from CSL Behring, and grants, personal fees and non-financial support from LFB, outside the submitted work.

Conflict of interest: A.M. Turner reports consultancy fees from CSL Behring, grants from Grifols Biotherapeutics, the Alpha 1 Foundation, the ATS Foundation and the Chest Foundation, grants, conference attendance and fees for clinical trial work as investigator paid to her institution from Arrowhead Inc., and fees for work as study investigator paid to her institution from Boehringer, outside the submitted work.

Conflict of interest: N.G. McElvaney reports grants and personal fees from CSL Behring, grants, and personal fees and non-financial support from Grifols, outside the submitted work.

Conflict of interest: E. Derom has nothing to disclose.

Conflict of interest: E. van 't Wout has nothing to disclose.

Conflict of interest: M. Canotilho has nothing to disclose.

Conflict of interest: M. Sucena has nothing to disclose.

Support statement: The physician survey and development of this manuscript was funded by CSL Behring. Medical writing assistance was provided by Steven Foster (Meridian HealthComms Ltd, Plumley, UK), funded by CSL Behring. Funding information for this article has been deposited with the Crossref Funder Registry.